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When Should Unmet Need Trump Inadequate Data?

 
• By Bridget Silverman

The Esbriet review exemplifies FDA’s eternal struggle between requiring adequate evidence of efficacy and giving patients with deadly diseases a chance at treatment.

FDA has proven time and again that it will extend considerable regulatory flexibility to drugs for serious unmet medical needs and orphan diseases, but it decided to issue a “complete response” letter for InterMune Inc.’s Esbriet (pirfenidone) for idiopathic pulmonary fibrosis in 2010 – and IPF patients had to wait four more years for approval of the first drugs to treat the rare and fatal lung condition.

Review documents show that FDA struggled with the decision, and that it had an unexpected ally in choosing to wait for better evidence of efficacy: patient advocates.

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More from United States

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• By Sarah Karlin-Smith

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Clinical Trials Nonprofit Fights for Access Despite DEI Crack-Down

 
• By Sue Sutter

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More from North America

US FDA Cell-Gene Therapy Head Says Agency Has Revived Stalled Programs

 
• By Sarah Karlin-Smith

CBER's Nicole Verdun wants rare disease sponsors with stalled treatments to circle back because the FDA's evolved thinking on clinical trial designs may offer another opportunity for cast away products.

FDA’s FY 2026 Budget Request Lacks New Policy Proposals

 
• By Sue Sutter

The White House requested $6.8bn for the FDA, down 3.9% from the current funding level, but does not propose any legislative changes. In previous years, the agency used the budget process to seek statutory fixes specific to generic drugs and shortages.

User Fee-Funded Staff Would Drop In FY 2026 US FDA Budget

 
• By Derrick Gingery

The reductions across medical product programs in the agency’s budget request include hundreds of positions funded by user fees, which may foreshadow its upcoming user fee restructuring strategy.